PB2012: DESCRIBING TREATMENT PATTERNS IN PATIENTS WITH MYELODYSPLASTIC SYNDROME ACCORDING TO IPSS-R RISK CATEGORY
Authors: Sorcha Corry, Arushi Bamrara, Núria Lara, Daniel Cuadras, Federica Centorrino, Ricardo Viana
Journal: HemaSphere (2023)

Topic: 10. Myelodysplastic syndromes - Clinical Background: Myelodysplastic syndrome (MDS) is a heterogeneous entity of hematologic disorders leading to a substantial range of prognosis for patients. Patients’ management and treatment goals vary according to their prognostic risk, per the revised International Prognostic Scoring System (IPSS-R), and individual characteristics. Patients at lower risk mainly receive supportive care whereas those at higher risk receive high-intensity therapy. Aims: The study aimed to assess real-world treatment patterns of MDS patients by their risk and age. Methods: An online survey was conducted in France, UK, Italy, and Canada amongst a panel of 139 haemato-oncologists who regularly manage MDS patients. Each panelist reported anonymous patient-level data for 3-6 patients, at least one for each pre-defined category: low risk (LR-MDS) including very low or low IPSS-R, intermediate risk (IR-MDS) corresponding to intermediate IPSS-R, and high risk (HR-MDS) including high or very high IPSS-R at the time of diagnosis. All patients must had received at least one line (1L) of therapy. Demographics, health status, and treatment pathway were collected for patients diagnosed between 2016 and 2021. Descriptive statistics were reported per risk category and age. Results: Data for 588 patients were collected; 59.4% were males and 56.5% were >70 years old at diagnosis. Patients’ distribution per risk category was: 43.3% HR, 23.0% IR and 33.7% LR-MDS. Mean follow-up duration was 22.1 months (LR, 26.9 months; IR, 24.1 months; HR, 17.2 months). Overall, 439 patients (74.7%) received only 1L of therapy, 136 patients (23.1%) a second line (2L), and 13 patients (2.2%) received a third line or more (+3L). No patient received more than four lines of treatment. Of the 439 patients that received only a 1L, 60.4% were >70 years of age. In the LR-MDS group, 18.2% patients received more than 1L of therapy (+1L); of them 36.1% were ≤70 years. Amongst patients with IR-MDS, 35.6% received +1L, of them, 56.2% were ≤70 years. In the HR-MDS group, 25.5% received +1L, 64.6% of them were ≤70 years. Among all patients, best supportive care (BSC) was the most frequent treatment for 1L (n=409; 69.6%), HMAs for 2L (n=60; 40.3%) and other therapies for 3L (n=7; 53.8%), venetoclax among them, used by 5 patients (38.5%). BSC was the most frequent treatment for LR-MDS patients as 1L and 2L treatment with 87.4% and 50.0% of patients, respectively. Hypomethylating agents (HMAs) were the second most used treatment in both lines (1L: 11.6%; 2L: 30.6%) (Table 1). Similar results were obtained for IR-MDS patients, with 78.5% and 43.8% being treated with BSC at 1L and 2L, respectively. HMAs remained the second most frequent treatment (1L: 36.3%; 2L: 35.4%) among these patients. For HR-MDS patients, the most frequent treatment was HMAs in both, 1L (70.6%) and 2L (49.2%), while BSC was the second most frequent treatment in both lines of therapy (1L: 51.0%; 2L: 75.0%). Only 18 patients received a 3L of therapy, with BSC being most used in LR-MDS (3 out of 4 patients) while venetoclax being most commonly used in IR-MDS (2 out of 5 patients) and HR-MDS groups (2 out of 4 patients). Venetoclax was used mainly by HR-MDS patients with 7.5% (n=19), 21.5% (n=14) and 50.0% (n=2) of patients receiving it in 1L, 2L and 3L, respectively.Summary/Conclusion: The majority of patients with MDS older than 70 years old received only 1L, while patients with IR or HR-MDS, and ≤ 70 years old, received more lines of therapy. The most common treatment in 1L was BSC for LR and IR-MDS, and HMAs for HR-MDS. Off-label use of venetoclax was mainly observed in HR-MDS patients. Keywords: Myelodysplastic syndrome, Treatment